Enhanced CRISPR to study health and disease in all cell types

Professor Ludovic Vallier, Dr Alessandro Bertero, and others from the University of Cambridge and Wellcome Trust Sanger Institute have developed a more efficient and inducible way of editing genes using CRISPR. Their single-step system is freely available and works in every cell of the body and at every stage of development. The new approach will have wide usability in cardiovascular and cancer research, tissue regeneration, and developmental biology.

The teams have developed two complementary methods: sOPTiKO is a knock-out system that turns off genes by disrupting the DNA, and sOPTiKD is a knock-down system that silences the action of genes by disrupting the RNA.

Using these two methods, scientists can turn off or silence genes in any cell type, at any stage of a cell’s development from stem cell to fully differentiated adult cell. These systems will allow researchers world-wide to rapidly and accurately explore the changing role of genes as the cells develop into tissues such as liver, skin or heart, and discover how this contributes to health and disease.

The sOPTiKO and sOPTiKD methods allow scientists to silence the activity of more than one gene at a time, so researchers are now able to investigate the role of whole families of related genes by knocking down the activity of all of them at once.

In addition, the freely available system allows experiments to be carried out far more rapidly and cheaply. sOPTiKO is highly flexible so that it can be used in every tissue in the body without needing to create a new system each time. sOPiTKD allows vast improvements in efficiency: it can be used to knock down more than one gene at a time. Before, to silence the activity of three genes, researchers had to knock down one gene, grow the cell line, and repeat for the next gene, and again for the next. Now it can do it all in one step, cutting a nine-month process down to just one to two months.

This study was published in Development. To find out more, please follow the above link to the original publication, or go to the University website.